OrkambiKIDS: Impact of CFTR-modulation with Lumacftor/Ivacaftor on the intestinal and respiratory microbiome and microbial metabolites in children aged 2 to 12 with cystic fibrosis

OrkambiKIDS is a clinical phase IV study designed to monitor treatment with Lumacftor/Ivacaftor of children aged 6 to 12 with cystic fibrosis (CF) homozygous for the F508del mutation. We aimed to assess changes in the intestinal and respiratory microbiome and metabolome in correlation with CF-typical biomarkers from stool, sputum and blood plasma during 24 months of study period at 9 different timepoints.